Title:
SIRNA FOR INHIBITING PCSK9 GENE EXPRESSION AND MODIFIER THEREOF AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/089486
Kind Code:
A1
Abstract:
An siRNA for inhibiting PCSK9 gene expression and a modifier thereof and the use thereof. Provided is an siRNA for inhibiting PCSK9 gene expression, which comprises a sense strand and an antisense strand; the sense strand and/or the antisense strand have a length in the range of 19-30 nucleotides; the sense strand and the antisense strand can be complementary to form a double-stranded RNA; and the complementary region forming the double-stranded RNA has a length in the range of 15-30 nucleotides. The siRNA molecules provided have a high inhibitory activity and a high stability, and have a better hepatic targeting effect and ability to promote cell endocytosis; and the ligand-modified siRNA molecules can enter target cells and target tissues without transfection reagents, reducing the negative effects of transfection reagents, such as cell or tissue toxicity. The ligand-modified siRNA molecules provide potential for a PCSK9 gene-mediated disease targeting therapy.
Inventors:
QIN YINLIN (CN)
SU MEI (CN)
DUAN CHUNXIAO (CN)
ZHANG PEIZHUO (CN)
SU MEI (CN)
DUAN CHUNXIAO (CN)
ZHANG PEIZHUO (CN)
Application Number:
PCT/CN2021/126724
Publication Date:
May 05, 2022
Filing Date:
October 27, 2021
Export Citation:
Assignee:
JIANGSU CAREPHAR PHARMACEUTICAL CO LTD (CN)
International Classes:
C12N15/113; A61K31/7105; A61K48/00; A61P35/00
Domestic Patent References:
| WO2019204021A1 | 2019-10-24 | |||
| WO2009114475A2 | 2009-09-17 |
Foreign References:
| CN104854242A | 2015-08-19 | |||
| CN109957565A | 2019-07-02 | |||
| CN110446784A | 2019-11-12 | |||
| CN101484588A | 2009-07-15 | |||
| EP2667197A1 | 2013-11-27 | |||
| US20050245475A1 | 2005-11-03 |
Other References:
LANDMESSER ULF, HAGHIKIA ARASH, LEITER LAWRENCE A, WRIGHT R SCOTT, KALLEND DAVID, WIJNGAARD PETER, STOEKENBROEK ROBERT, KASTELEIN : "Effect of inclisiran, the small-interfering RNA against proprotein convertase subtilisin/kexin type 9, on platelets, immune cells, and immunological biomarkers: a pre-specified analysis from ORION-1", CARDIOVASCULAR RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 117, no. 1, 1 January 2021 (2021-01-01), GB , pages 284 - 291, XP055925949, ISSN: 0008-6363, DOI: 10.1093/cvr/cvaa077
Attorney, Agent or Firm:
JEEKAI&PARTNERS (CN)
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